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Diabetes Research Gene Editing

Could Advancements in Gene Editing Reverse Type 1 Diabetes?

Gene therapy is not a new approach when it comes to treating type 1 diabetes. Scientists have been experimenting with many different options in order to stimulate the body to once again produce its own insulin and reduce or eliminate the need for insulin injections. However, some of the problems that scientists often encounter when introducing new cells into the body are that patients typically require immunosuppressant drugs which can lead to a variety of complications, the body rejects the cells over time, or the cells stop working. Finding a long-term, effective solution has been challenging.

Scientists are making strides in their efforts, though. A recent study examined the potential of using the gene-editing tool CRISPR to correct genetic mutations and create induced pluripotent stem cells that can be transformed into pancreatic beta cells. In mouse models, after the new cells were injected, mice achieved normoglycemia within a week and maintained this status for at least six months.

This approach has not yet been tested in humans, however, because it comes with its own set of challenges. First, the study was done using cells from patients with Wolfram syndrome, a condition that causes diabetes and deafness. This condition can be pinpointed to a single genetic mutation, whereas type 1 diabetes cannot. Type 1 diabetes has been tied to multiple gene mutations, as well as environmental factors. Gene-editing would have to be personalized for each individual, which could take a lot of time.

In addition, it could take billions of cells to effectively reverse diabetes in a patient, and generating this massive number of cells could take months, so it could end up being a long process to treat even one person. Plus, scientists are not entirely sure where the best place to transplant these cells is yet. They must find the spot where they will be most beneficial and able to carry out their intended purpose.

Another study using CRISPR technology is being conducted by a different group of researchers and is focused on using stem cells from the human cell line rather than from individual patients. This would make it easier to produce mass quantities of cells in a shorter period of time. It also would not require scientists to correct specific genetic mutations. CRISPR would be used to edit cells to prevent them from being attacked and destroyed by the body’s immune system.

A challenge with these approaches is that there are a lot of questions and regulations when it comes to gene-editing and using CRISPR on human subjects. Clinical trials are still in very early stages. Studies involving induced pluripotent stem cells are also relatively new in the United States. There is still a lot of work, research, and testing that needs to be done before gene-editing therapy could potentially be used on humans.

Diabetes Research Connection (DRC) will continue to follow these advancements and what they could mean for future diabetes treatment. DRC supports early-career scientists in contributing valuable discoveries and information of their own to the field by providing critical research funding. All projects funded by the DRC are focused on the prevention, treatment, and cure of type 1 diabetes, as well as minimizing complications and improving the quality of life for individuals living with the disease. Learn more and support these efforts by visiting https://diabetesresearchconnection.org.

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DNA Strand

Using Gene Editing as a Potential Type 1 Diabetes Treatment

It has been more than a decade since scientists began experimenting with CRISPR gene-editing technology to alter DNA sequences and gene function. This tool allows scientists to correct mutations or defects in genes and manipulate them to treat or prevent certain diseases. This technology has also been used with crops. Researchers are still exploring this tool’s potential and ethical use, but many studies have been conducted thus far using it in different ways.

A recent study examines the use of CRISPR-Cas9 in the treatment of diabetes. Scientists at Washington University in St. Louis corrected a mutation in the WFS1 gene which causes Wolfram syndrome, of which diabetes is one symptom. Then, they used CRISPR-Cas9 to edit human-induced pluripotent stem cells and target their differentiation into pancreatic beta cells. This creates an abundance of fully functional beta cells to be used in conjunction with gene therapy.

When the altered beta cells were transplanted into diabetic mice, blood glucose levels dropped and glycemic control was maintained for at least six months. Scientists are exploring whether this process can be used to effectively reverse or stop type 1 diabetes by editing a patient’s own beta cells. In addition, the abundance of cells created means that more testing can occur to develop specific medications or therapies to treat the disease.

More research is needed before gene editing can potentially be used as an approved treatment for type 1 diabetes, but researchers continue to learn more. Diabetes Research Connection (DRC) is interested to see what this technology may mean for the future of diabetes treatment and management and how it could evolve. Though not involved with this study, the DRC is committed to supporting research around type 1 diabetes and provides early-career scientists with critical funding for novel, peer-reviewed studies. To learn more about current projects and how to help, visit https://diabetesresearchconnection.org.

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OUR PROJECTS

See our approved research projects and campaigns.

Role of the integrated stress response in type 1 diabetes pathogenesis
In individuals with type 1 diabetes (T1D), the insulin-producing beta cells are spontaneously destroyed by their own immune system. The trigger that provokes the immune system to destroy the beta cells is unknown. However, accumulating evidence suggest that signals are perhaps first sent out by the stressed beta cells that eventually attracts the immune cells. Stressed cells adapt different stress mitigation systems as an adaptive response. However, when these adaptive responses go awry, it results in cell death. One of the stress response mechanisms, namely the integrated stress response (ISR) is activated under a variety of stressful stimuli to promote cell survival. However, when ISR is chronically activated, it can be damaging to the cells and can lead to cell death. The role of the ISR in the context of T1D is unknown. Therefore, in this DRC funded study, we propose to study the ISR in the beta cells to determine its role in propagating T1D.
Wearable Skin Fluorescence Imaging Patch for the Detection of Blood Glucose Level on an Engineered Skin Platform
zhang
A Potential Second Cure for T1D by Re-Educating the Patient’s Immune System
L Ferreira
Validating the Hypothesis to Cure T1D by Eliminating the Rejection of Cells From Another Person by Farming Beta Cells From a Patient’s Own Stem Cells
Han Zhu
Taming a Particularly Lethal Category of Cells May Reduce/Eliminate the Onset of T1D
JRDwyer 2022 Lab 1
Can the Inhibition of One Specific Body Gene Prevent Type 1 Diabetes?
Melanie
Is Cholesterol Exacerbating T1D by Reducing the Functionality and Regeneration Ability of Residual Beta Cells?
Regeneration Ability of Residual Beta Cells
A Call to Question… Is T1D Caused by Dysfunctionality of Two Pancreatic Cells (β and α)?
Xin Tong
Novel therapy initiative with potential path to preventing T1D by targeting TWO components of T1D development (autoimmune response and beta-cell survival)
flavia pecanha